#developing #rarediseasetherapies is hard. Developing them for #rarepediatricdiseases is even harder. How do we bring more therapies to market for #children? How do we develop the right FDA strategies -and what impact will the new (ish) #FDA guidances have? How do we ensure we have the right incentives including pediatric rare disease priority review vouchers #PRVs- and how do #BPCA and #PREA work together as ‚carrot‘ and ‚stick‘? Thank you Laurie Conklin, MD , Christine Lee-Flemming, PhD, PharmD, Dionna Green and Thomas Miller for a fascinating and thought-provoking conversation at #Bio2024! My key take-away: we want ( need!) to include children earlier, but we need to get this right and have a strong scientific justification to inform the study design, and to get there we need the sophistication to leverage all the tools in the tool belt including #clinicaltrial simulation studies and model-informed trial design. A brave new world is upon us - if we can make the leap successfully!
Thank you for attending!
Absolutely agree.
Life Science Executive, Board Director & Strategic Advisor
6moCould not agree more Karin - thanks for attending!